rAAV-compatible MiniPromoters for restricted expression in the brain and eye View Full Text


Ontology type: schema:ScholarlyArticle      Open Access: True


Article Info

DATE

2016-12

AUTHORS

Charles N. de Leeuw, Andrea J. Korecki, Garrett E. Berry, Jack W. Hickmott, Siu Ling Lam, Tess C. Lengyell, Russell J. Bonaguro, Lisa J. Borretta, Vikramjit Chopra, Alice Y. Chou, Cletus A. D’Souza, Olga Kaspieva, Stéphanie Laprise, Simone C. McInerny, Elodie Portales-Casamar, Magdalena I. Swanson-Newman, Kaelan Wong, George S. Yang, Michelle Zhou, Steven J. M. Jones, Robert A. Holt, Aravind Asokan, Daniel Goldowitz, Wyeth W. Wasserman, Elizabeth M. Simpson

ABSTRACT

BACKGROUND: Small promoters that recapitulate endogenous gene expression patterns are important for basic, preclinical, and now clinical research. Recently, there has been a promising revival of gene therapy for diseases with unmet therapeutic needs. To date, most gene therapies have used viral-based ubiquitous promoters-however, promoters that restrict expression to target cells will minimize off-target side effects, broaden the palette of deliverable therapeutics, and thereby improve safety and efficacy. Here, we take steps towards filling the need for such promoters by developing a high-throughput pipeline that goes from genome-based bioinformatic design to rapid testing in vivo. METHODS: For much of this work, therapeutically interesting Pleiades MiniPromoters (MiniPs; ~4 kb human DNA regulatory elements), previously tested in knock-in mice, were "cut down" to ~2.5 kb and tested in recombinant adeno-associated virus (rAAV), the virus of choice for gene therapy of the central nervous system. To evaluate our methods, we generated 29 experimental rAAV2/9 viruses carrying 19 different MiniPs, which were injected intravenously into neonatal mice to allow broad unbiased distribution, and characterized in neural tissues by X-gal immunohistochemistry for icre, or immunofluorescent detection of GFP. RESULTS: The data showed that 16 of the 19 (84 %) MiniPs recapitulated the expression pattern of their design source. This included expression of: Ple67 in brain raphe nuclei; Ple155 in Purkinje cells of the cerebellum, and retinal bipolar ON cells; Ple261 in endothelial cells of brain blood vessels; and Ple264 in retinal Müller glia. CONCLUSIONS: Overall, the methodology and MiniPs presented here represent important advances for basic and preclinical research, and may enable a paradigm shift in gene therapy. More... »

PAGES

52

References to SciGraph publications

  • 2012-10. Beyond knockouts: cre resources for conditional mutagenesis in MAMMALIAN GENOME
  • 2015-12. Protein tyrosine phosphatase receptor type R is required for Purkinje cell responsiveness in cerebellar long-term depression in MOLECULAR BRAIN
  • 2009-03. An anatomic gene expression atlas of the adult mouse brain in NATURE NEUROSCIENCE
  • 2012-02. Novel adeno-associated viral vectors for retinal gene therapy in GENE THERAPY
  • 2013-12. Non-coding-regulatory regions of human brain genes delineated by bacterial artificial chromosome knock-in mice in BMC BIOLOGY
  • 2011-02. Charting a course for genomic medicine from base pairs to bedside in NATURE
  • 2012-12. Predicting tissue specific cis-regulatory modules in the human genome using pairs of co-occurring motifs in BMC BIOINFORMATICS
  • 2014-12. Optogenetic astrocyte activation modulates response selectivity of visual cortex neurons in vivo in NATURE COMMUNICATIONS
  • 2015-10. Gene therapy returns to centre stage in NATURE
  • 2015-10. Paradigm shifts in genomics through the FANTOM projects in MAMMALIAN GENOME
  • 2008-12. Identification of a set of genes showing regionally enriched expression in the mouse brain in BMC NEUROSCIENCE
  • 2012-10. The mammalian gene function resource: the international knockout mouse consortium in MAMMALIAN GENOME
  • 2012-08. Gene therapy for Leber congenital amaurosis: advances and future directions in GRAEFE'S ARCHIVE FOR CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY
  • 2015-12. Nr2e1 regulates retinal lamination and the development of Müller glia, S-cones, and glycineric amacrine cells during retinogenesis in MOLECULAR BRAIN
  • 2016. Tissue-Specific Promoters in the CNS in GENE THERAPY FOR NEUROLOGICAL DISORDERS
  • 2009-05. Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS in GENE THERAPY
  • 2012-09. An integrated encyclopedia of DNA elements in the human genome in NATURE
  • 2009-01. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes in NATURE BIOTECHNOLOGY
  • 2014-10. Gene Therapy for the Nervous System: Challenges and New Strategies in NEUROTHERAPEUTICS
  • 2007-01. Genome-wide atlas of gene expression in the adult mouse brain in NATURE
  • 2014-12. Characterization of dsRed2-positive cells in the doublecortin-dsRed2 transgenic adult rat retina in HISTOCHEMISTRY AND CELL BIOLOGY
  • 2012-04. Metazoan promoters: emerging characteristics and insights into transcriptional regulation in NATURE REVIEWS GENETICS
  • 2011-05. Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges in NATURE REVIEWS GENETICS
  • Identifiers

    URI

    http://scigraph.springernature.com/pub.10.1186/s13041-016-0232-4

    DOI

    http://dx.doi.org/10.1186/s13041-016-0232-4

    DIMENSIONS

    https://app.dimensions.ai/details/publication/pub.1015877525

    PUBMED

    https://www.ncbi.nlm.nih.gov/pubmed/27164903


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